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Clinical Trial With Riluzole in Spinocerebellar Ataxia Type 2 (ATRIL) (ATRIL)

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ClinicalTrials.gov Identifier: NCT03347344
Recruitment Status : Completed
First Posted : November 20, 2017
Last Update Posted : June 21, 2021
Sponsor:
Information provided by (Responsible Party):
Assistance Publique - Hôpitaux de Paris

Tracking Information
First Submitted Date  ICMJE September 21, 2017
First Posted Date  ICMJE November 20, 2017
Last Update Posted Date June 21, 2021
Actual Study Start Date  ICMJE January 17, 2018
Actual Primary Completion Date December 14, 2020   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures  ICMJE
 (submitted: November 17, 2017)
Change in Ataxia symptoms (Scale for the Assessment and Rating of Ataxia (SARA)) [ Time Frame: at 12 months. ]
To compare the proportion of patients with Scale for the Assessment and Rating of Ataxia (SARA) improvement (decrease) of at least one point from baseline to 12 months
Original Primary Outcome Measures  ICMJE Same as current
Change History
Current Secondary Outcome Measures  ICMJE
 (submitted: November 17, 2017)
  • Change in Ataxia symptoms (Composite Cerebellar Functional Severity (CCFS) score) [ Time Frame: at 12 months ]
    To compare the difference of the CCFS score (Composite Cerebellar Functional Severity Score) from baseline at 12 months. A decrease is expected in the intervention group.
  • Change in extracerebellar symptoms (Inventory of Non-Ataxia Signs (INAS)) [ Time Frame: at 12 months ]
    To compare the difference of the extracerebellar symptoms (INAS, Inventory of Non-Ataxia Signs) by showing decrease in the INAS count from baseline at 12 months
  • 12 months survival [ Time Frame: at 12 months ]
    To compare survival of the patients between the two treatment groups at 12 months
Original Secondary Outcome Measures  ICMJE Same as current
Current Other Pre-specified Outcome Measures Not Provided
Original Other Pre-specified Outcome Measures Not Provided
 
Descriptive Information
Brief Title  ICMJE Clinical Trial With Riluzole in Spinocerebellar Ataxia Type 2 (ATRIL)
Official Title  ICMJE Multicenter, Randomized, Double Blind, Placebo Controlled Clinical Trial With Riluzole in Spinocerebellar Ataxia Type 2
Brief Summary

ATRIL is a multi-centric, double-blind randomized, two-arm controlled study. 42 SpinoCerebellar Ataxia type 2 (SCA2) patients, both gender, at least 18 years of age will be included.

Riluzole 50 mg will be administered (per os) twice a day, versus one group with placebo for 12 months.

Riluzole (Rilutek®) is a benzothiazole drug, market approved, for Amyotrophic Lateral Sclerosis (ALS). It delays the onset of ventilator-dependence or tracheostomy in selected patients and may increase survival.

Scale for the Assessment and Rating of Ataxia (SARA) will be used at M0, M6 and M12. To assess primary criterion, the percentage of patients with a decrease of at least 1 point of the SARA score between the inclusion visit, and Visit 3 (Months 12) will be calculated.

Detailed Description Inherited cerebellar ataxias are genetically heterogeneous neurological disorders. They are characterized by ataxic gait and cerebellar dysarthria that progresses over time with loss of ambulation and speech. The mutations by expansions of CAG triplets in the genes ATXN1 (SCA1), ATXN 2 (SCA2), 3 (SCA3), CACNA1A (SCA6), ATXN 7 (SCA7), and TBP (SCA17) are responsible for 50% of hereditary forms There is no curative or preventive treatment. This phase III study is a multi-centric, double-blind randomized, two-arm controlled study (one group with 50 mg Riluzole twice a day versus one group with placebo), to measure the efficacy of treatment with riluzole in SCA2 patients during 12 months. Amelioration is defined by a 1 point decrease of the SARA score.
Study Type  ICMJE Interventional
Study Phase  ICMJE Phase 3
Study Design  ICMJE Allocation: Randomized
Intervention Model: Parallel Assignment
Intervention Model Description:
Multi-centric, double-blind randomized, two-arm controlled study (one group with 50 mg riluzole twice a day versus one group with placebo), during one year
Masking: Triple (Participant, Care Provider, Investigator)
Masking Description:

Treatments will be presented in numbered boxes, labeled for this study according to the Good Manufacturing Practices by the General Agency of Equipment and Health Products (AGEPS).

Each numbered box will consist of 6 months of treatment: 20 blister packs of 20 active or placebo tablets

Primary Purpose: Treatment
Condition  ICMJE Spinocerebellar Ataxia Type 2
Intervention  ICMJE
  • Drug: Riluzole
    50 mg will be administered (per os) twice a day
  • Drug: Placebo
    50 mg will be administered (per os) twice a day
Study Arms  ICMJE
  • Experimental: RILUZOLE
    Riluzole PMCS 50 mg is presented as a round, biconvex, 8 mm diameter nearly white film-coated tablet. The tablets will be held under a blister of 20 tablets.
    Intervention: Drug: Riluzole
  • Placebo Comparator: PLACEBO
    The placebo PMCS 50 mg is presented as a round, biconvex, 8 mm diameter nearly white film-coated tablet matching the appearance of the Riluzole used in this study
    Intervention: Drug: Placebo
Publications * Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruitment Information
Recruitment Status  ICMJE Completed
Actual Enrollment  ICMJE
 (submitted: November 17, 2017)
42
Original Estimated Enrollment  ICMJE Same as current
Actual Study Completion Date  ICMJE December 14, 2020
Actual Primary Completion Date December 14, 2020   (Final data collection date for primary outcome measure)
Eligibility Criteria  ICMJE

Inclusion Criteria:

  • Genetically diagnosed SCA2 (CAG triplet in ATXN2 ≥ 33)
  • At least 18 years of age
  • Signature of informed consent
  • Covered by social security
  • SARA score ≥ 5 and ≤ 26
  • Age at onset ≤ 50 years old

Exclusion Criteria:

  • Treated with riluzole prior to the study
  • Hepatotoxic medication
  • Hypersensitivity to the active substance or to any of the excipients
  • Serious systemic illnesses or conditions known for enhancing the side effects of riluzole
  • Contraindications for MRI examination
  • Participation in another therapeutic trial (3 months exclusion period)
  • Pregnancy or breastfeeding
  • Non abstinence or absence of effective contraception for women
  • Inability to understand information about the protocol
  • Persons deprived of their liberty by judicial or administrative decision
  • Adult subject under legal protection or unable to consent
  • Other ataxic syndromes than SCA2
Sex/Gender  ICMJE
Sexes Eligible for Study: All
Ages  ICMJE 18 Years and older   (Adult, Older Adult)
Accepts Healthy Volunteers  ICMJE No
Contacts  ICMJE Contact information is only displayed when the study is recruiting subjects
Listed Location Countries  ICMJE France
Removed Location Countries  
 
Administrative Information
NCT Number  ICMJE NCT03347344
Other Study ID Numbers  ICMJE P160927J
2017-001481-23 ( EudraCT Number )
Has Data Monitoring Committee No
U.S. FDA-regulated Product
Studies a U.S. FDA-regulated Drug Product: No
Studies a U.S. FDA-regulated Device Product: No
IPD Sharing Statement  ICMJE Not Provided
Responsible Party Assistance Publique - Hôpitaux de Paris
Study Sponsor  ICMJE Assistance Publique - Hôpitaux de Paris
Collaborators  ICMJE Not Provided
Investigators  ICMJE
Principal Investigator: DURR Alexandra, PU-PH ASSISTANCE PUBLIQUE HÖPITAUX DE PARIS
PRS Account Assistance Publique - Hôpitaux de Paris
Verification Date June 2021

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP